“All stakeholders need to challenge their assumptions about the clinical trial paradigm and collaborate in new ways”

The COVID-19 pandemic has created renewed appreciation for the tremendous impact healthcare and pharmaceutical innovation has on the health and daily lives of society. Public-private partnerships to develop vaccines and therapeutics in response to the pandemic have significantly raised public awareness about clinical trials and global clinical trial infrastructure. Established clinical development paradigms in which one therapy is studied in one trial have been challenged by newer approaches in which novel collaborations and innovation in clinical trial design can generate more timely and reliable evidence to advance the most promising new therapies to patients.

The central vision of EU-PEARL, from its inception, was to develop a new “Integrated Research Platform” (IRP) paradigm for clinical development that is more patient-centric and more efficient. IRPs bring together understanding of the disease (through registries, longitudinal natural history studies and real world data), cohorts of trial-ready patients, and a platform trial capable of studying multiple therapies simultaneously.  Platform trials utilize specialized statistical approaches and establish a network of sites and investigators – a re-useable clinical trial infrastructure – focused on finding solutions for patients in a particular disease.  As promising therapies advance through the platform trial, the IRP framework ensures the regulatory pathway is established.

Success will require leading academic medical centers and other institutions to take on new or expanded roles in clinical research. This may include becoming the sponsor of a platform trial, an important role with specific legal and regulatory responsibilities. Ultimately, all stakeholders – patients, industry, regulators, and investigators – will need to challenge their assumptions about the clinical trial paradigm and collaborate in new ways to realize the shared goal of bringing safe and effective treatment options to patients.

Within the EU-PEARL consortium, we’ve seen significant learning across consortium members and an open exchange of ideas. We’ve also seen strong collaboration with external stakeholders and innovators on whose work we are building from across Europe, the UK, and the US.  Slightly more than one year into the project, we are already collaborating to define how to sustain the development of IRPs to ensure maximal impact for patients, even beyond the scope of the EU-PEARL project itself.

By placing the patient at the center of our Integrated Research Platform concept, all stakeholders can unite around a common purpose and realize the benefits of a clinical development paradigm designed to address unmet needs of patients, and society.

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